One of the world's top experts in coronary heart disease is spearheading a new gene editing upstart out to transform the field
As head of the Center for Human Genetic Research at Massachusetts General Hospital and the Broad’s Cardiovascular Disease Initiative, Sekar Kathiresan has occupied a singular position as one of the world’s leading experts on the connection between genetics and coronary heart disease. He’s tracked how people dealt a bad genetic hand — and the elevated risks that come with it — can limit inherent dangers by lifestyle changes, and pondered over the effects of daily drugs used to treat mass patient groups. And he’s reached a simple conclusion:
None of it is really working.
In particular, none of that is anywhere near as useful as the genetic mutations that he’s seen that confer a lower risk of dying and cardio events. Particularly the individuals who carry mutations “which break either of two genes — APOC3 or ANGPTL3 — rapidly clear triglyceride-rich lipoproteins from the circulation” and protect them from heart attack.
Now, he’s making a professional leap to see if he and a squad of investigators at a newly launched biotech can dramatically change the imperfect status quo through gene editing.
“Imagine,” he tells me, “an injection administered once in life that safely confers lasting protection.”
No more pills. No fleeting commitments to healthy lifestyles that can’t stretch past January. But a widespread and durable sharing of the same health benefits he’s seen in the very, very few. That’s the dream.
Today, Kathiresan is stepping down from his lofty academic roles and making his debut as CEO of Verve Therapeutics, which is taking its place in the hotbed of gene therapy work around Cambridge, MA. The small team of 10 — soon to double in size — may not come close to rivaling the big biotechs that occupy the streets in and around Harvard and MIT. But relatively few can claim the same kinds of connections in the realms of drug science.
Another cardio genetics expert, Penn’s Kiran Musunuru and Harvard professor J Keith Joung, who co-founded gene therapy pioneer Editas, are on board as scientific co-founders. There’s an alliance with Beam Therapeutics, the upstart next-gen gene editing outfit founded by Feng Zhang, one of 3 scientists widely credited with ushering in the CRISPR revolution that has armed researchers around the world with effective tools to accomplish their work. And Verily will help work on the nanoparticles they plan to use for delivery.
Burt Adelman, the former EVP of R&D at Biogen, will chair the board, which includes the Broad’s chief data officer, Anthony Philippakis.
Beam will provide some of the tech, and has an option to step in on future commercialization. Verve has also nailed down CRISPR patents, including Cas9 and Cas12a (Cpf1), from the Broad Institute and Harvard University.
And they have $58.5 million in cash to do their work from GV (you probably still think of them as Google Ventures), which is stepping in with ARCH Venture Partners, F-Prime Capital, and Biomatics Capital to form the original syndicate.
To be successful, the Verve team under Kathiresan will not just have to demonstrate their approach can safely work, but also that it ultimately can be done on a mass basis in economic terms. That’s a tall order, but they do have some advantages, perhaps most noticeably the advances the groundbreakers have made at the FDA, says the scientist.
“Gene editing has the potential to completely transform the treatment paradigm for the disease,” says Musunuru. “Preclinical studies conducted in the field, including work done in my lab, have shown the promise of gene editing to safely reduce cholesterol and other coronary artery disease risk factors.”
So far, gene editing in the legitimate R&D world has been centered on the painstaking advances of a handful of programs aimed at rare diseases. And Verve will start with its own rare ailments, targeting patient populations with the highest unmet medical need. But Kathiresan isn’t dropping his prestigious academic roles to search for marginal gains. He wants to tackle the whole threat on a worldwide basis.
That, officially, starts at Verve today.